Gene Therapists Celebrate a Decade of Progress
It has taken many years, but researchers may have reached a prized goal in gene therapy: lowering the risk of uncontrolled bleeding in patients with hemophilia. At a meeting last week, researchers reported that six patients who received a virus engineered to carry a gene for a blood-clotting protein called factor IX needed fewer transfusions of the protein for as long as 18 months; some didn't require any transfusions. One patient developed an immune response to the viral vector, but this side effect was successfully treated with drugs.
Sangamo gene therapy shows promise in reducing HIV
An early stage trial of Sangamo BioSciences Inc's HIV treatment found that the gene therapy reduced levels of the virus and even eliminated it in one patient with a naturally occurring gene mutation. The very small Phase 1 trial tested the SB-728-T gene therapy, which is designed to disrupt the CCR5 gene used by HIV to infect cells of the immune system. If shown to be safe and effective, the treatment could end the need for the antiretroviral drugs now used to keep the virus that causes AIDS in check by suppressing viral replication in the blood.
Bubble Boys Treated With Gene Therapy Still Healthy After 9 Years
It’s been a big month for gene therapy: first a breakthrough for leukaemia last week, now today scientists announced they’ve successfully treated kids with “bubble boy” disease. We’ve heard this before — back in 2000 gene therapy successfully treated the same immune disease, but by 2007 five out of the 10 boys had developed leukaemia (ironically, the disease successfully treated in the study announced last week), and one died. This time, the researchers smartly waited NINE YEARS to declare success with 14 out of 16 boys. It’s hard to argue with nearly a decade of good health.
Genetically modified cells destroy leukaemia tumours
Scientists for the first time have used gene therapy to successfully destroy cancer tumours in patients with advanced disease - a goal that has taken 20 years to achieve. Researchers at the University of Pennsylvania engineered patients' own pathogen-fighting T-cells to target a molecule found on the surface of leukaemia cells.
The altered T-cells were grown outside of the body and infused back into patients suffering from late-stage chronic lymphocytic leukaemia (CLL), which affects the blood and bone marrow and is the most common form of leukaemia.
Evolution: A View from the 20th Century
Evolution: A View from the 20th Century describes the many ways that cells reorganize their own genomes in the course of evolution. This genome restructuring involves processes such as symbiogenesis, horizontal DNA transfer, and natural genetic engineering within the cell. These natural processes, as homologous recombination, transposition and site-specific recombination have already done, can provide models for artificial genetic engineering. Hopefully, increased focus on the evolutionary capabilities of living cells will expand the range of technologies available for gene therapy.
Survey on Risk Assessment in Clinical Trials
Please consider participating in the following survey, which is being conducted by gene therapy and ethics researchers from the University of Sydney. This research aims to investigate how gene therapy researchers take account of considerations of risk when making decisions about the design and conduct of gene therapy clinical trials. The survey is completely anonymous and takes approximately 10-15 minutes to complete.
Amsterdam Molecular Therapeutics Receives Opinion on Glybera Marketing Authorisation Application
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera® (alipogene tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency ("LPLD"). Following a recent meeting with the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), AMT has been notified that, at this time, Glybera is not approvable.
Subsequent to a review of the CHMP's letter, AMT believes that Glybera can receive a positive opinion subject to generating additional data from existing patients. AMT has therefore decided to ask for a re-examination of the clinical data package.
New approach to cancer vaccines proves successful in early studies
University of Leeds researchers, funded by Cancer Research UK, have used a library of DNA to create a vaccine that could be used to treat cancer, according to a study published in Nature Medicine. Before now, ‘gene therapy’ vaccines have often delivered just one gene to stimulate the immune system. It produces a protein, called an antigen, which activates the immune system to destroy cancer cells. It has been difficult to develop successful cancer vaccines because each tumour has specific proteins and identifying the right antigens has been a huge challenge
A new start for gene therapy for “bubble boy” disease: First U.S.-treated patient doing well
Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns.
After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. Jeremías had to stop attending nursery school, for fear he’d bring home an infection his baby brother might catch. When Agustín’s relatives came to help out, they had to change their clothes and wash their hands, and couldn’t enter Agustín’s room.
Monkey Vaccine Has Promise For Eventual Human HIV Vaccine
A vaccine has been developed that protects non-human primates (monkeys) from SIV (Simian Immunodeficiency Virus), the monkey equivalent of HIV. Researchers reported in the journal Nature that this breakthrough could eventually lead to a vaccine for humans, protecting them from HIV. Louis Picker, M.D., from the Vaccine and Gene Therapy Institute (VGTI) in Oregon, and team produced a vaccine that programs monkeys' immune systems to respond more rapidly to the presence of SIV. Researchers from the International AIDS Vaccine Initiative and the National Cancer Institute-Fredick were also involved in this research.
Gene Therapy Will be a Vital Part of Future Health Care
More than 60% of the people are of the opinion that gene therapy will be a vital part of future health care. Only 3% think that there is no future for gene therapy as it causes more problems than produce solutions. These are the results of an internet poll among the visitors of Gene Therapy Net.
Moving gene therapy into high gear
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes.
International collaboration has been critical. Children’s Hospital Boston is one of the founding members of the Transatlantic Gene Therapy Consortium (TAGTC), a new collaboration that seeks to facilitate a more rapid advancement of this technology for treating human diseases. It was initiated shortly after the first trials of gene therapy for X-linked Severe Combined Immunodeficiency (X-SCID) (in both Paris and London) reported leukemia as a serious side effect. The TAGTC was formed to address this setback, developing safer gene therapy reagents, sharing the costs of their development, and then implementing new gene therapy trials for rare diseases across multiple international sites.
First Clinical Trial of Gene Therapy for Pain Shows Substantial Pain Relief for Patients
In the first clinical trial of gene therapy for treatment of intractable pain, researchers from the University of Michigan Department of Neurology observed that the treatment appears to provide substantial pain relief. In a study published online in the Annals of Neurology last week, the researchers showed that the novel agent NP2 is safe and well-tolerated. In addition, measures of pain in the treated patients suggested that NP2 may provide a substantial analgesic effect.
EU Clinical Trials Register goes live
Public online register gives access to information on clinical trials. The EU Clinical Trials Register (https://www.clinicaltrialsregister.eu) was launched today by the European Medicines Agency (EMA). The online register gives for the first time public access to information on interventional clinical trials for medicines authorised in the 27 EU Member States and Iceland, Liechtenstein and Norway. The database also allows the public to search for information on clinical trials authorised to be carried out outside the EU if these trials are part of a paediatric investigation plan. See also Clinical Trials Databases.
Gene therapy a reality for Parkinson's
A groundbreaking study headquartered in Detroit has found that half the Parkinson's patients treated with gene therapy (NLX-P101) had lasting, "clinically meaningful improvements" without serious side effects, the Henry Ford Health System announced Tuesday.
The study is the first time gene therapy has proven successful in Parkinson's patients and could substantially improve the lives of one million Americans with the degenerative disorder, said Dr. Peter LeWitt, director of movement disorders and lead author of the study.
NVGT Embraces Cell Therapy with Name Change
The Dutch Society of Gene Therapy (NVGT) has officially changed its name last Friday to the Netherlands Society of Gene and Cell Therapy (NVGCT), a change intended to better reflect the intimate relationship between the two fields.
The NVGCT is dedicated to promoting basic and clinical research in gene therapy and cell therapy within the Netherlands. By connecting scientists, clinicians, patients, public and government we wish to contribute to the development of gene and cell therapy for various diseases. The society aims to facilitate the exchange of information and technology, improve communication towards the public and provide education within the professional community.
Zinc finger gene therapy produces HIV-resistant CD4 T-cells
Gene therapy that interferes with co-receptors on the surface of T-cells can protect these cells from HIV infection, representing a potential first step toward achieving a "functional cure," researchers reported at the 18th Conference on Retroviruses and Opportunistic Infections (CROI), taking place this week in Boston.
HIV uses two different surface co-receptors, CCR5 and CXCR4, to enter CD4 T-cells. If the co-receptors are blocked or disrupted, the virus is unable to enter cells. Two presentations on Monday looked at using gene therapy to create cells that lack these receptor proteins and therefore are protected from infection.
New Anti-HIV Gene Therapy Makes T-Cells Resistant to HIV Infection
An innovative genetic strategy for rendering T-cells resistant to HIV infection without affecting their normal growth and activity is described in a paper published in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.
A team of researchers from Japan, Korea, and the U.S. developed an anti-HIV gene therapy method in which a bacterial gene called mazF is transferred into CD4+ T-cells. The MazF protein is an enzyme (an mRNA interferase) that destroys gene transcripts, preventing protein synthesis. The design of this mazF gene therapy vector ensures that synthesis of the MazF protein is triggered by HIV infection. When HIV infects treated T lymphocytes, MazF is induced, blocking HIV replication and, essentially, making the T-cells HIV resistant.
Gene therapy to fix memory loss in Alzheimer's patients study
A latest research by scientists at the Gladstone Institute of Neurological Disease (GIND) in San Francisco has revealed that a new gene therapy technique might hold the key to Alzheimer's cure. The study found that increasing the levels of EphB2, a protein responsible for regulation of neural communication in the brain, alleviated symptoms of long-term memory loss in mice.
Cognitive processes such as learning and memory retention require effective communication between brain cells called neurons. This communication involves release of chemicals that stimulate cell surface receptors present on the nerve cells. The process, called neurotransmission, is impaired by amyloid plaques, which build-up to abnormally high levels in the brains of Alzheimer's patients and are believed to be the root cause the disease.
The Use of Gene Therapy in Treating Retinitis Pigmentosa and Dry Age-related Macular Degeneration (AMD)
Irv Arons recently completed a comprehensive treatise on the use of gene therapy for treating retinitis pigmentosa (RP) and the dry form of age-related macular degeneration (AMD). The report introduces RetroSense Therapeutics and its efforts to develop a gene therapy treatment to restore vision in those suffering from RP (first) and dry AMD (second). Included are sections on alternative therapies for both RP and AMD, including retinal implants (for RP), stem cells, and sustained release drugs and a few other options. In addition, there is a short writeup on other applications of gene therapy in ophthalmology.
NIH RAC and CliniGene Co-host a Scientific Symposium on 'Retroviral and Lentiviral Vectors for Long-Term Gene Correction: Clinical Challenges in Vector and Trial Design'
In past safety symposia, the NIH Recombinant DNA Advisory Committee (RAC) reviewed clinical and molecular data concerning leukemias caused by insertional mutagenesis at or near oncogenes in two trials for X-linked Severe Combined Immunodeficiency Disease (X-SCID) involving transduction of CD34+ hematopoetic stem cells by retroviral vectors. Since these discussions, investigators have reported advances in the field, including clinical benefits in trials studying other clinical indications (e.g., adenosine deaminase deficient-SCID, adrenoleukodystrophy). In addition, research has focused on alternative vectors, either lentiviral vectors or modified retroviral vectors designed to decrease the risk of enhancer-mediated insertional mutagenesis.
Given these developments, as well as recent reports of myelodysplasia in a trial for chronic granulomatous disease and the finding of a relative clonal population of cells in a trial for thalassemia, the RAC and the European Network for the Advancement of Clinical Gene Transfer and Therapy (CliniGene) are holding this symposium (December 9-10, 2010, Bethesda).
First patient being treated by Stem cell therapy
An unnamed person suffering from a spinal cord injury has become the first patient to receive Stem Cell Therapy for his/her injuries and is being treated at the Shepherd Center, a 132-bed hospital in Atlanta specializing in spinal cord and brain injuries. The treatment is being sponsored by Geron Corp. of Menlo Park, California. Without releasing any information about the patient or the nature of the injury, doctors have said that the treatment will be one of first to test the effectiveness and safety of such a process and tests will be conducted to see whether the treatment restores sensation or enables the patient to regain movement.
Leading gene therapy researcher retracts more papers
Savio Woo of the Mount Sinai School of Medicine in New York has retracted two more papers this week, the blog Retraction Watch reported today. That brings Woo’s total number of retractions this year to six, following an investigation that found evidence of scientific misconduct committed by two of his postdocs.
Four of the six retracted papers, including the two most recently pulled, describe a technique that harnesses a viral enzyme called ‘phiBT1 integrase’ to shuttle genes into the mouse genome, particularly in the liver. The retractions for all of these papers merely cite “data irregularities”. Michele Calos, a geneticist at Stanford University, says her lab was unable to reproduce the phiBT1 work and found numerous factual errors in the published procedures. Also, Woo’s team claimed that phiBT1 only inserted into the genome at the regions between genes—a key advantage of the technique because it reduced the odds that an insertion would disrupt the function of a normal gene. But that didn’t hold up in her lab, Calos notes.
Gene Therapy Stops Blood Disorder in Patient, Ending Need for Transfusions
A 21-year-old man with a blood disorder who needed monthly transfusions to survive since he was 3 had his condition halted by a gene therapy procedure. The man suffered from beta thalassemia, a common genetic disease that reduces red blood cell production. He was treated in Paris in 2007 and hasn’t needed a transfusion in two years, according to a study published today in the journal Nature. The therapy, being developed by closely held Bluebird Bio of Cambridge, Massachusetts, is the latest in a series of successful gene treatments. Bluebird, backed by four venture capital firms and Genzyme Corp., the world’s largest maker of drugs for rare genetic diseases, plans to treat nine more patients who have thalassemia or sickle cell anemia. The company also is developing a gene therapy that has helped patients with a rare vision disorder to see.
Gene Doping Detectable With a Simple Blood Test
German scientists from Tübingen and Mainz have developed a blood test that can reliably detect gene doping even after 56 days. Scientists at the universities in Tübingen and Mainz have developed a test that can provide conclusive proof of gene doping. "For the first time, a direct method is now available that uses conventional blood samples to detect doping via gene transfer and is still effective if the actual doping took place up to 56 days before," Professor Perikles Simon, MD, PhD from Johannes Gutenberg University Mainz, Germany explained. "This represents a relatively low-cost method of detecting several of the most common doping genes," Simon stated in the presentation of the process.
Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations
The FDA will convene a one-day workshop (November 2, 2010 - 8:00 AM-5:30 PM) to facilitate an exchange of information about best practices in conducting cell and gene therapy clinical trials in pediatric populations. Cellular and gene therapies are the subject of great interest as novel products that potentially may improve the lives of patients by restoring lost function and modifying the nature and course of diseases. However, these therapies are not without risks: indeed, the novelty of these products contributes to their real and potential risks. Conducting clinical trials with novel products in pediatric patients requires special scrutiny to ensure that the rights of subjects are protected and that potential risks and benefits are appropriately balanced.
The purpose of the workshop is to gather information from Institutional Review Boards (IRBs), gene and cellular therapy clinical researchers, and other stakeholders regarding best practices related to cell and gene therapy clinical trials in pediatric populations, as well as challenges and considerations in the review of these clinical trials.
New Hope Against Melanoma
By taking a different approach, scientists have developed the next promising cancer treatment: targeted gene therapy. Skin cancer is scary, and it's tricky. If caught early it is easily treatable, but if not, it's potentially deadly. Even the best dermatologists can't spot everything. Targeted gene therapy is the latest treatment recognized by the New England Journal of Medicine. The new approach is proving fruitful when it comes to patients with the deadliest form of skin cancer: metastasized melanoma.
Gene Therapy to Treat Epilepsy a Step Closer
Current antiepileptic drugs (AEDs) have many side-effects, among others slowing down brain activity, which in turn reduces patients' ability to react. These side-effects could be eliminated if genes that counteract seizures could be introduced into the brain. Professor Merab Kokaia at Lund University in Sweden has obtained promising results in animal experiments.
CliniGene Competitive Call for Additional Project Partners
Programme title: CliniGene, (Contract number LSHB-CT-2006-018933), the European Network of Excellence for the Advancement of Clinical Gene Transfer and Therapy.
CliniGene seeks additional partners to enter its Joint Programme Activity on the topics:
Topic 1. Non-viral integrating systems for clinical gene therapy and development of human iPS, in particular transposon based technology;
Topic 2. AAV for in vivo gene therapy, in particular mechanisms leading to tight transcription regulation;
Topic 3. Technology transfer to SMEs for: (i) systems which have benefited from common evaluation and fine-tuning inside the NoE; (ii) a technology that is believed to complement the CliniGene current portfolio with view to performing side to side comparisons with gene transfer technologies already evaluated within CliniGene.
Study Shows Hope for Gene Therapy
Researchers have launched a new gene-therapy trial for children with a rare disease known as "bubble boy syndrome," reflecting fresh hopes that the strategy of delivering working genes can be used to treat many intractable ailments.
In the new study, sponsored in the U.S. by investigators at Children's Hospital Boston and expected to open at five sites around the world, scientists plan to enroll 20 boys with SCID-X1, which stands for severe combined immunodeficiency, X-linked—a genetic condition that affects boys and leaves them unable to fight germs. Without treatment, which is currently possible only by bone-marrow transplantation, most children die before age one.